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Adeno Associated Virus Aav Vectors In Gene Therapy
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Book Synopsis Adeno-Associated Virus (AAV) Vectors in Gene Therapy by : Kenneth I. Berns
Download or read book Adeno-Associated Virus (AAV) Vectors in Gene Therapy written by Kenneth I. Berns and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its se quence determined and its regulation adequately characterized. Second, a suitable vector for the delivery of a good copy of the affected gene must be available. For a vector to be of use several attributes are highly desirable: these include ability to carry the intact gene (although this may be either the genomic or the cDNA form) in a stable form, ability to introduce the gene into the desired cell type, ability to express the introduced gene in an appropriately regulated manner for an extended period of time, and a lack of toxicity for the recipient. Also of concern is the frequency of cell transformation and, in some cases, the ability to introduce the gene into nondividing stem cells. Sev eral animal viruses have been tested as potential vectors, but none has proven to have all the desired properties described above. For example, retroviruses are difficult to propagate in sufficient titers, do not integrate into nondividing cells, and are of concern because of their oncogenic properties in some hosts and because they integrate at many sites in the genome and, thus, are potentially insertional mutagens. Additionally, genes introduced by retroviral vectors are frequently expressed for relatively short periods of time. A second virus used as a vector in model systems has been adenovirus (Ad).
Book Synopsis Fields Virology: Emerging Viruses by : Peter M. Howley
Download or read book Fields Virology: Emerging Viruses written by Peter M. Howley and published by Lippincott Williams & Wilkins. This book was released on 2020-02-11 with total page 2597 pages. Available in PDF, EPUB and Kindle. Book excerpt: Now in four convenient volumes, Field’s Virology remains the most authoritative reference in this fast-changing field, providing definitive coverage of virology, including virus biology as well as replication and medical aspects of specific virus families. This volume of Field’s Virology: Emerging Viruses, 7th Edition covers recent changes in emerging viruses, providing new or extensively revised chapters that reflect these advances in this dynamic field.
Book Synopsis Viral Vectors for Gene Therapy by : Fredric P. Manfredsson
Download or read book Viral Vectors for Gene Therapy written by Fredric P. Manfredsson and published by . This book was released on 2019 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.
Book Synopsis Immunopharmacology by : Manzoor M. Khan
Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.
Book Synopsis AAV Gene Therapy: Immunology and Immunotherapeutics by : Jose Martinez-Navio
Download or read book AAV Gene Therapy: Immunology and Immunotherapeutics written by Jose Martinez-Navio and published by Frontiers Media SA. This book was released on 2022-02-09 with total page 187 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme
Book Synopsis Viral Vectors for Gene Therapy by : Curtis A. Machida
Download or read book Viral Vectors for Gene Therapy written by Curtis A. Machida and published by Springer Science & Business Media. This book was released on 2008-02-02 with total page 591 pages. Available in PDF, EPUB and Kindle. Book excerpt: Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The “Notes” section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors.
Book Synopsis Challenges in Delivery of Therapeutic Genomics and Proteomics by : Ambikanandan Misra
Download or read book Challenges in Delivery of Therapeutic Genomics and Proteomics written by Ambikanandan Misra and published by Elsevier. This book was released on 2010-09-09 with total page 686 pages. Available in PDF, EPUB and Kindle. Book excerpt: Delivery of therapeutic proteomics and genomics represent an important area of drug delivery research. Genomics and proteomics approaches could be used to direct drug development processes by unearthing pathways involved in disease pathogenesis where intervention may be most successful. This book describes the basics of genomics and proteomics and highlights the various chemical, physical and biological approaches to protein and gene delivery. Covers a diverse array of topics from basic sciences to therapeutic applications of proteomics and genomics delivery Of interest to researchers in both academia and industry Highlights what’s currently known and where further research is needed
Book Synopsis Holland-Frei Cancer Medicine by : Robert C. Bast, Jr.
Download or read book Holland-Frei Cancer Medicine written by Robert C. Bast, Jr. and published by John Wiley & Sons. This book was released on 2017-03-10 with total page 2004 pages. Available in PDF, EPUB and Kindle. Book excerpt: Holland-Frei Cancer Medicine, Ninth Edition, offers a balanced view of the most current knowledge of cancer science and clinical oncology practice. This all-new edition is the consummate reference source for medical oncologists, radiation oncologists, internists, surgical oncologists, and others who treat cancer patients. A translational perspective throughout, integrating cancer biology with cancer management providing an in depth understanding of the disease An emphasis on multidisciplinary, research-driven patient care to improve outcomes and optimal use of all appropriate therapies Cutting-edge coverage of personalized cancer care, including molecular diagnostics and therapeutics Concise, readable, clinically relevant text with algorithms, guidelines and insight into the use of both conventional and novel drugs Includes free access to the Wiley Digital Edition providing search across the book, the full reference list with web links, illustrations and photographs, and post-publication updates
Book Synopsis Muscle Gene Therapy by : Dongsheng Duan
Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.
Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog
Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne
Book Synopsis Immune responses to AAV vectors, from bench to bedside by : Federico Mingozzi
Download or read book Immune responses to AAV vectors, from bench to bedside written by Federico Mingozzi and published by Frontiers Media SA. This book was released on 2015-06-30 with total page 97 pages. Available in PDF, EPUB and Kindle. Book excerpt: The recent wave of clinical studies demonstrating long-term therapeutic efficacy highlights the enormous potential of gene therapy as an approach to the treatment of inherited disorders and cancer. While in recent years lentiviral vectors have dominated the field of ex vivo gene therapy in man, adeno-associated virus (AAV) vectors have become the platform of choice for the in vivo gene delivery, both local and systemic. Despite the achievements in the clinic however, a number of hurdles remain to be overcome in gene therapy, these include availability of scalable vector production systems, potential issues associated with insertional mutagenesis, and concerns related to immunogenicity of gene therapeutics. For AAV vectors, clinical trials showed that immunity directed against the vector could either prevent transduction of a target tissue or limit the duration of therapeutic efficacy. Initial observations in the context of a gene therapy trial for hemophilia spurred over a decade efforts by gene therapists and immunologists to understand the mechanism and identify factors that contribute to AAV’s immunogenicity, including the prevalence of B cell and T cell immunity to wild type AAV in humans and the interaction of AAV vectors with the innate and adaptive immune system. Despite a number of important contributions in particular in the more recent past, our knowledge on the immunology of gene transfer is still rudimental; this is partly due to the fact that the basic understanding of the complex balance between tolerance and immunity to an antigen, key aspect of gene transfer with AAV, keeps evolving rapidly. However, continuing work towards a better definition of the interaction of viral vectors with the immune system has led to significant advances in the knowledge of the factors influencing the outcome of gene transfer, such as the vector dose, the immune privilege of certain tissues, and the induction of tolerance to an antigen. A better understanding of the structure-function relationship of the viral capsid has boosted the development of novel immune-escape vector variants. In addition, novel immunomodulatory strategies were established to prevent or reduce anti-capsid immunity have been developed and are being tested in preclinical models and in clinical trials. Together, these advances are bringing us closer to the goal of achieving safe and sustained therapeutic gene transfer in humans. In this research topic, a collection of Original Research and Review Articles highlights critical aspects of the interaction between gene AAV vectors and the immune system, discussing how these interactions can be either detrimental or constitute an advantage, depending on the context of gene transfer, and providing tools and resources to better understand the issue of immunogenicity of AAV vectors in gene transfer.
Book Synopsis Adenoviral Vectors for Gene Therapy by : David T. Curiel
Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 870 pages. Available in PDF, EPUB and Kindle. Book excerpt: Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials
Book Synopsis Capillary Gel Electrophoresis by : Andras Guttman
Download or read book Capillary Gel Electrophoresis written by Andras Guttman and published by Newnes. This book was released on 2021-12-04 with total page 391 pages. Available in PDF, EPUB and Kindle. Book excerpt: Capillary Gel Electrophoresis and Related Microseparation Techniques covers all theoretical and practical aspects of capillary gel electrophoresis. It also provides an excellent overview of the key application areas of nucleic acid, protein and complex carbohydrate analysis, affinity-based methodologies, micropreparative aspects and related microseparation methods. It not only gives readers a better understanding of how to utilize this technology, but also provides insights into how to determine which method will provide the best technical solutions to particular problems. This book can also serve as a textbook for undergraduate and graduate courses in analytical chemistry, analytical biochemistry, molecular biology and biotechnology courses. Covers all theoretical and practical aspects of capillary gel electrophoresis Excellent overview of the key applications of nucleic acid, protein and complex carbohydrate analysis, affinity-based methodologies, micropreparative aspects and related microseparation methods Teaches readers how to use the technology and select methods that are ideal for fundamental problems Can serve as a textbook for undergraduate and graduate courses in analytical chemistry, analytical biochemistry, molecular biology and biotechnology courses
Book Synopsis Gene Therapy for Diseases of the Lung by : Kenneth Brigham
Download or read book Gene Therapy for Diseases of the Lung written by Kenneth Brigham and published by CRC Press. This book was released on 1997-04-10 with total page 460 pages. Available in PDF, EPUB and Kindle. Book excerpt: This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems.
Book Synopsis Adeno-associated Virus Vectors by : Michael J. Castle
Download or read book Adeno-associated Virus Vectors written by Michael J. Castle and published by . This book was released on 2019 with total page 426 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Engineering of Novel Adeno-Associated Virus Vectors for Gene Therapy Applications by : Jorge Luis Santiago Ortiz
Download or read book Engineering of Novel Adeno-Associated Virus Vectors for Gene Therapy Applications written by Jorge Luis Santiago Ortiz and published by . This book was released on 2016 with total page 102 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy – the introduction of genetic material into cells and tissues of interest for a therapeutic purpose – has emerged as a very promising treatment for many diseases. Recent advances in genomics and proteomics, coupled with the advent of genome editing technologies, have generated an immense pool of potential nucleic acid cargoes that could be delivered as therapies for a wide array of diseases, ranging from monogenic disorders to cancer. However, before such therapies can be successful, a major hurdle must be overcome: the development of gene-carrying vehicles – also referred to as vectors – that can safely, efficiently, and specifically deliver those therapeutic payloads to the desired cells. The goal of this dissertation was therefore to address a major need in the field: the development of improved gene delivery vectors. To date, more than 2,000 clinical trials employing gene transfer have taken place, establishing the safety of a number of vectors. Non-viral vectors can be easily produced at a large-scale and are amenable to the engineering of their chemical and physical properties via chemical modifications, but they suffer from a low delivery efficiency and cell toxicity. On the other hand, viral vectors harness the highly evolved mechanisms that viruses have developed to efficiently recognize and infect cells and offer several advantages that make them suitable candidates for use in gene delivery, both for therapeutic application and as tools for biological studies. In fact, gene therapy has enjoyed increasing success in clinical trials for numerous disease targets in large part due to the gene delivery capabilities viral vectors. Vectors derived from viruses have been used in the majority (over 68%) of gene therapy clinical trials to date, and the most frequently used have been based on adenovirus, retrovirus, vaccinia virus, herpesvirus, and adeno-associated virus (AAV). AAV vectors are non-pathogenic and can transduce numerous dividing and non-dividing cell types. Because of these characteristics, AAV vectors have been utilized for gene therapy in various tissues. The amino acid composition of the viral capsid affects tropism (tissue specificity), cell receptor usage, and susceptibility to anti-AAV neutralizing antibodies – properties that influence efficacy in therapeutic gene delivery. However, AAV vectors can still encounter formidable impediments to efficacious gene delivery, including poor transduction (infection and expression of delivered gene) of some cell types, off-target transduction, difficulties with biological transport barriers, and potential risks associated with the integration of their genetic load. Extensive engineering of the AAV capsid promises to overcome these delivery challenges and improve numerous clinically relevant properties. To this end, the overarching goal of my work in the Schaffer Laboratory, which is presented in this thesis dissertation, was to advance current gene delivery methods through the engineering and characterization of novel adeno-associated virus vectors for gene therapy and research applications. To access new viral capsid sequences with potentially enhanced infectious properties and to gain insights into AAV’s evolutionary history, we computationally designed and experimentally constructed an ancestral AAV capsid library. We performed selection for infectivity on the library, studied the resulting amino acid distribution, and characterized the selected variants, which yielded viral particles that were broadly infectious across multiple cell types. Ancestral variants displayed higher thermostability than modern (extant) natural AAV serotypes, a property that makes them promising templates for protein engineering applications, including directed evolution. Additionally, some variants displayed high in vivo infectivity on a mouse model, highlighting their potential for gene therapy. Motivated by the success of directed evolution in the engineering of proteins with novel or enhanced properties, I worked in the engineering of AAV vectors for gene delivery to glioblastoma multiforme (GBM), a highly aggressive type of brain cancer. For this, I conducted directed evolution to select AAV variants with selective localization to and infectivity on GBM tumor cells and tumor initiating cells (TICs). Using an accurate GBM mouse model, I performed in vitro and in vivo selection, recovering viral particles that successfully trafficked to tumor cells and TICs in the brain after systemic administration to tumor-bearing animals. Following three rounds of in vivo selection, convergence was achieved upon several variants, the most abundant of which emerged from the ancestral reconstruction library. The selected variants are currently being characterized and assessed for their ability to deliver reporter and therapeutic genes, hopefully resulting in improved suppression of tumor progression compared to delivery with existing AAV serotypes. These novel vectors could enable new, potent therapies to treat GBM tumors and pave the way for engineering AAV vectors for other cancer targets. In summary, this dissertation presents work on the development and characterization of a novel AAV capsid library, as well as on the implementation of this and of other libraries towards the engineering of novel AAV variants with selective gene delivery properties for brain tumors. The work herein presented aims to advance both the field of AAV vector engineering as a whole and the specific application of AAV vectors towards next generation cancer therapies.
Book Synopsis Creating Enhanced Adeno-associated Viral Vectors for Gene Delivery Using Directed Evolution by : Narendra Maheshri
Download or read book Creating Enhanced Adeno-associated Viral Vectors for Gene Delivery Using Directed Evolution written by Narendra Maheshri and published by . This book was released on 2004 with total page 286 pages. Available in PDF, EPUB and Kindle. Book excerpt:
